The purpose of this proposal is to request partial support for the "9th Symposium on the Molecular Biology of Hematopoiesis". This four day symposium, which will be held in Genoa, Italy from June 24-27, 1995, will address molecular genetic aspects of hematopoiesis, with special emphasis on the development of therapeutic strategies for hematopoietic diseases and other diseases in which hematopoietic cells may be targets. Specifically, the funds requested will be used to support a scientific session within the symposium entitled: "Hematopoietic Stem Cell Gene Therapy for Lysosomal Storage Disorders". Many lysosomal storage diseases (e.g., Gaucher disease, Type B Niemann-Pick disease, Mucopolysaccharidosis Type VII) are considered excellent candidates for hematopoietic stem cell gene therapy. In fact, human stem cell gene therapy trials are about to be undertaken for the most common lysosomal disorder, Gaucher disease, and several other diseases are currently being considered. Thus, it is particularly timely and appropriate to include a scientific session within this hematopoietic symposium which deals specifically with the development of stem cell gene therapy for lysosomal diseases. The session will emphasize work on animal models since there are many excellent lysosomal animal models in which stem cell gene therapy is actively being developed (e.g., Mucopolysaccharidosis Types I, VI, VII, Niemann-Pick disease). The lectures currently planned for this session include: Dr. Mark Haskins (University of Pennsylvania), who will discuss the results of bone marrow transplantation in the lysosomal animal model systems, Dr. Steven Walkley (Albert Einstein College of Medicine), who will discuss the entrance of hematopoietic cells into the central nervous system and the feasibility of treating neurologic lysosomal storage diseases by stem cell gene therapy, Dr. Jan Visser (New York Blood Center), who will discuss optimization of protocols for gene transfer into rodent (mouse and rat) stem cells, Dr. Janis Abkowitz (University of Washington), who will discuss optimization of protocols for gene transfer into large animal (cat and dog) stem cells, Dr. John Wolfe (University of Pennsylvania), who will discuss in utero gene transfer in animal model systems, and Dr. Edward H. Schuchman (Mount Sinai School of Medicine), who wilt serve as the session organizer and speak on specific issues to be considered in treating lysosomal diseases by stem cell gene therapy. It is hoped that through this conference new ideas and approaches will emerge which will advance our understanding of lysosomal diseases and, specifically, advance our efforts to develop hematopoietic stem cell gene therapy for these devastating disorders. Moreover, this symposium will provide a valuable opportunity for scientists directly involved in the study of lysosomal storage diseases to interact with expert hematologists (including a large number of stem cell biologists) from throughout the world. In turn, these interactions will hopefully lead to productive collaborations and new research endeavors.